Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy

• ClinicalTrials.gov Identifier: NCT01847573
• Recruitment Status: Terminated
• First Posted: May 7, 2013
• Last Update Posted: September 3, 2020
• Sponsor: Processa Pharmaceuticals
• Information provided by (Responsible Party): Processa Pharmaceuticals

Tracking Information

• First Submitted Date: May 2, 2013
• First Posted Date: May 7, 2013
• Last Update Posted Date: September 3, 2020
• Study Start Date: May 2013
• Actual Primary Completion Date: March 30, 2016 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 6, 2013)

Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys [ Time Frame: 1 week ]

Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: May 6, 2013)

• Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys [ Time Frame: 1 week ]
  Halofuginone plasma concentrations
• Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks [ Time Frame: 4 weeks ]
  Safety profile by review of AEs, physical examination findings, clinical laboratory test results, and other diagnostic testing
• Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys [ Time Frame: 4 weeks ]
  Pharmacodynamic measures relevant to DMD pathology:

  • Pulmonary function
  • Motor function
  • Muscle composition
  • Biochemical and imaging markers

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
• Official Title: A Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-100 in Patients With Duchenne Muscular Dystrophy
• Brief Summary: The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD). The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with DMD. In this study, pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream will also be taken.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Duchenne Muscular Dystrophy

Intervention

Drug: HT-100

May be administered in either fed or fasted state

Other Name: halofuginone hydrobromide delayed-release tablet

Study Arms

• Experimental: Cohort 1: HT-100 tablet, Dose 1
  • Single dose administration: Dose 1
  • Multiple dose administration: Dose 1
  Intervention: Drug: HT-100
• Experimental: Cohort 2: HT-100 tablet, Dose 2
  • Single dose administration: Dose 2
  • Multiple dose administration: Dose 2
  Intervention: Drug: HT-100
• Experimental: Cohort 3: HT-100 tablet, Dose 3
  • Single dose administration: Dose 3
  • Multiple dose administration: Dose 3
  Intervention: Drug: HT-100
• Experimental: Cohort 4a: HT-100 tablet, Dose 4
  • Single dose administration: Dose 4
  • Multiple dose administration: Dose 4
  Intervention: Drug: HT-100
• Experimental: Cohort 4b: HT-100 tablet, Dose 5
  * Multiple dose administration: Dose 5
  Intervention: Drug: HT-100
• Experimental: Cohort 5: HT-100 tablet, Dose 6
  * Multiple dose administration: Dose 5
  Intervention: Drug: HT-100

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Terminated
• Actual Enrollment (submitted: April 21, 2017): 17
• Original Estimated Enrollment (submitted: May 6, 2013): 30
• Actual Study Completion Date: March 30, 2016
• Actual Primary Completion Date: March 30, 2016 (Final data collection date for primary outcome measure)

Eligibility Criteria

Main Inclusion Criteria:

• Ambulatory or non-ambulatory
• Diagnosis of DMD with confirmation of minimal to no dystrophin
• Corticosteroid naive or on therapy for at least 12 months (stable dose and regimen)

Main Exclusion Criteria:

• Recent, substantial change in use of cardiac medications or medications affecting muscle function
• Inability to undergo magnetic resonance imaging (MRI)
• Significantly compromised cardio-respiratory function
• Prior treatment with another investigational product in past 6 months

Sex/Gender

• Sexes Eligible for Study: Male

• Ages: 6 Years to 20 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01847573
• Other Study ID Numbers: HALO-DMD-01; HALO ( Other Identifier: Akashi Therapeutics )
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Processa Pharmaceuticals
• Original Responsible Party: Akashi Therapeutics
• Current Study Sponsor: Processa Pharmaceuticals
• Original Study Sponsor: Akashi Therapeutics
• Collaborators: Not Provided

Investigators

• Study Director: Diana M Escolar, MD; Akashi Therapeutics

• PRS Account: Processa Pharmaceuticals
• Verification Date: March 2019